Allele-specific silencing Duchenne muscular dystrophy LMNA Connective tissue Cancer biomarkers Myotubes Heart failure Muscle Titin Biological sciences Laminopathie Regeneration Butyrylcholinesterase Autophagosome maturation Skeletal muscle Biomarker Heart Clinical trial Joint laxity LGMD CSF protein Cancer Muscle biopsy Patient registry BiP AAV VECTOR Myopathy CMTX INPP5K Emery-Dreifuss muscular dystrophy Muscle MRI COL6A3 Collagen VI-related myopathies NGS collagen type VI congenital muscular dystrophy CMD limb-girdle muscular dystrophy LGMD muscular MRI neuromuscular disorders Maladies rares et orphelines Gene therapy Dystrophie musculaire Mutations Adult SMA Angiotensin-converting enzyme inhibitor C elegans Lamins C2C12 LMNA gene Lamin A/C nuclei Cardiac conduction system Nuclear envelope Acetyltransferase Therapy GNE Lamin A/C LMNA gene Neuromuscular diseases Muscular dystrophy MD LMNA-related congenital muscular dystrophy Exome Congenital muscular dystrophy Alternative splicing Next generation sequencing Diagnosis Lamin A/C Hypermobile EDS A-type lamin Muscular dystrophy Dilated cardiomyopathy Myogenesis COL6A1 Myologie Emerin Base de données FAIR Allele-specific silencing therapy Centronuclear myopathy Angiotensin-converting enzyme inhibitors Maladies rares Cardiomyopathy CRISPR Rare diseases Allele‐specific silencing therapy Laminopathies Errance diagnostique COVID-19 Becker muscular dystrophy Cardiology COL1A1 AAV Mouse Laminopathy BVES Calcium handling IPSC A-type lamins Dystrophine POPDC1 Dynamin 2 Actionability Treatment Actionable gene Ehlers‐Danlos Syndrome Becker muscular dystrophy BMD Duchenne muscular dystrophy DMD miRNA nNOS Treatment delay Myopathies RNA interference Rare neuromuscular diseases